Pipeline – Overview of the target diseases

Our company carefully evaluates and selects target diseases for exosome (EV) formulations.

Disease Overview[1]	ARDS is a severe respiratory failure caused by acute lung injury. Symptoms include sudden breathlessness, difficulty breathing, and severe shortness of breath. Mortality rate is 35-46%.[1] Treatment involves respiratory management, such as oxygen therapy and mechanical ventilation, and drug therapy. However, current pharmacological interventions, including steroids, have limited efficacy and lack strong evidence for improving survival rates.[3] Therefore, there is a pressing need for the development of new, effective drugs that can significantly reduce mortality and improve patient outcomes in ARDS.
Market Size[2]	The global market for ARDS therapeutics is projected to reach approximately $3.7 billion in 2024 and $4.9 billion in 2029, with an average annual growth rate of 5.7%.[2]
Reasons for Targeting	ARDS has a high mortality rate, but no drugs significantly improve survival. [3] Mesenchymal stem cells (MSCs) are known to have anti-inflammatory effects and have shown potential in improving ARDS survival rates in some clinical trials. Our MSC-derived EXP01 demonstrated higher anti-inflammatory effects in disease model mice compared to using MSCs as intact cells. Given the lack of effective treatments and the promising results of MSC-based clinical trials, targeting ARDS with EXP01 presents a significant opportunity to win the market.

Disease Overview[4]	IPF is a progressive disease causing lung fibrosis and difficulty breathing. Median survival after diagnosis is 3-5 years. Approved anti-fibrotic drugs have significant side effects.[1,4]
Market Size[2]	The global market for IPF therapeutics is expected to grow from $5.2 billion in 2024 to $7.3 billion by 2029, with an average annual growth rate of 7.0%.[6]
Reasons for Targeting	Current IPF treatments, such as pirfenidone and nintedanib, have limited efficacy and significant side effects, leading to poor patient outcomes. MSCs have demonstrated potential in reducing inflammation and fibrosis, showing promise in clinical trials. Our candidate EXP01, derived from MSCs using our proprietary technology, has shown superior anti-fibrotic effects in preclinical studies. By targeting IPF with EXP01, we aim to provide a more effective and safer therapeutic option to address the unmet needs of IPF patients.

Background	Infertility affects approximately 1 in 6 couples globally. As more women delay childbearing, age-related declines in oocyte quality and quantity have made natural conception increasingly difficult. In vitro fertilization (IVF) is a major treatment option for infertility and is widely practiced, with millions of cycles conducted annually worldwide. However, success rates remain limited, particularly for patients aged 35 and older. Poor embryo development and reduced implantation rates pose significant challenges in this population. As a result, there is an increasing demand for new technologies and additives that can consistently improve embryo quality.
Market Size[6]	The global market for IVF consumables and devices was valued at USD 2.3 billion in 2023, and is projected to reach USD 4.9 billion by 2028, growing at a CAGR of 13.6%.[7] Other estimates suggest the market could reach USD 8.9 billion by 2033, driven by delayed pregnancies and increased IVF adoption.[8]
Why We Target This Indication	Using our proprietary technology, we have successfully developed a naive-type extracellular vesicle (EV) product, EXP02, derived from human umbilical cord tissue. These EVs are rich in bioactive components essential for developing oocytes and sperm. In preclinical studies using mouse models, EXP02 significantly improved early embryo development, suggesting its strong potential as a solution to enhance the success rates of IVF treatments.

[1] The Japanese Respiratory Society, Japan Intractable Diseases Information Center, Crit Care. 2023 27:31
[2] Mordor intelligence 2023
[3] ARDS Clinical Practice Guidelines 2021
[4] The Japanese Respiratory Society, Japan Intractable Diseases Information Center
[5] Boston Consulting Group estimate (Ministry of Economy, Trade and Industry FY2023 Healthcare Industry Infrastructure Enhancement Promotion Project)
[6] Global Market Insights 2023
[7] MarketsandMarkets, “IVF Devices and Consumables Market – Global Forecast to 2028”
[8] Market.us, “Global In Vitro Fertilization Market”